ALXN1850: ALXN1850 will be administered via subcutaneous (SC) injection.
Placebo: Placebo will be administered via SC injection.
Study summary
The primary objective of this study is to assess the efficacy of ALXN1850 versus placebo on functional outcomes in adolescent and adult participants with HPP who have not previously been treated with asfotase alfa.
Eligibility
Sex
ALL
Min age
12 Years
Max age
130 Years
Healthy volunteers
No
Inclusion Criteria:
* Diagnosis of HPP documented in the medical records
* Must meet 1 of the following criteria:
1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a Clinical Laboratory Improvement Amendments (CLIA) or ISO 15189 certified laboratory (Section 8.7 )
2. Plasma PLP above the upper limit of normal (ULN) during the Screening Period (central or local laboratory results allowed per local regulations)
* Must meet 1 of the following criteria without a probably cause other than HPP:
1. Serum ALP activity below the age- and sex-adjusted normal range during the screening period as measured by the Central Laboratory
2. Two documented serum ALP activity results, at least 15 days apart, below the age- and sex-adjusted local laboratory normal range during the 24 months before the Day 1 Visit. Note: Local laboratories need to be CLIA or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion's approval.
* Two separate 6MWTs at below 85% of the predicted distance (for age, sex, weight, and height) during the Screening Period without a probable cause other than HPP
Exclusion Criteria:
* History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, neurological disorders, or any other disorders that are capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study intervention; or interfering with the interpretation of data as determined by the Investigator
* Diagnosis of primary or secondary hyperparathyroidism
* Hypoparathyroidism, unless secondary to HPP
* Any new fracture within 12 weeks before Day 1 (excluding pseudofractures)
* Planned surgical intervention which may impact the results of study assessments (in the opinion of the Investigator) during the Randomized Evaluation Period
* History of allergy or hypersensitivity to any ingredient contained in ALXN1850 or the placebo comparator (Table 9)
Primary outcome measure(s)
Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169) — Baseline, Day 169
Trial sites (65)
Facility
City
Region
Status
Research Site
Indianapolis
Indiana
Research Site
Garden City
New York
Research Site
Durham
North Carolina
Research Site
Columbus
Ohio
Research Site
Nashville
Tennessee
Research Site
Ciudad de Buenos Aires
Argentina
Research Site
Clayton
Australia
Research Site
Herston
Australia
Research Site
Parkville
Australia
Research Site
St Leonards
Australia
Research Site
Belo Horizonte
Brazil
Research Site
Brasília
Brazil
Research Site
Recife
Brazil
Research Site
Salvador
Brazil
Research Site
São Paulo
Brazil
Research Site
São Paulo
Brazil
Research Site
Calgary
Alberta
Research Site
Edmonton
Alberta
Research Site
Winnepeg
Manitoba
Research Site
Oakville
Ontario
Research Site
Trois-Rivières
Quebec
Research Site
Beijing
China
Research Site
Beijing
China
Research Site
Changsha
China
Research Site
Chengdu
China
Research Site
Nanchang
China
Research Site
Qingdao
China
Research Site
Shanghai
China
Research Site
Shenzhen
China
Research Site
Paris
France
Research Site
Poitiers
France
Research Site
Bad Reichenhall
Germany
Research Site
Berlin
Germany
Research Site
Bonn
Germany
Research Site
Hamburg
Germany
Research Site
Würzburg
Germany
Research Site
Ashkelon
Israel
Research Site
Ramat Gan
Israel
Research Site
Florence
Italy
Research Site
Milan
Italy
+ 25 more sites — see the full list on the official registry below.
More Alexion Pharmaceuticals, Inc. trials in the UK
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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